Invus Financial Advisors

City Therapeutics is a biopharmaceutical company focusing on engineering next-generation small-interfering RNAs (siRNAs) to enhance RNAi-based therapies. The company develops a pipeline of innovative RNAi therapeutics aimed at addressing various disease indications. City Therapeutics prioritizes improving and expanding RNAi technology to achieve better patient outcomes. The leadership team includes experienced scientists and industry professionals dedicated to advancing the field of RNA interference.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Syros Pharmaceuticals is a life sciences company that is focused on treating disease by mapping gene regulatory circuits and modulating the factors that regulate gene expression. Syros has pioneered world-leading gene control research and drug discovery capabilities with a proven ability to integrate disease biology and genomic data--a proficiency that is not well represented in pharmaceutical R&D. Central to the Syros approach is a proprietary platform of carefully integrated assay technologies, bioinformatics, and biologic insights developed by members of Syros' senior leadership. While this scientific approach has applications in many therapeutic areas, Syros has demonstrated success initially in oncology, where it may help address numerous unmet medical needs.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing gene therapies through its innovative Anellovector platform. This platform leverages the biology of the human commensal virome to create redosable and targetable DNA therapies that address several limitations of existing gene therapies, such as restricted access to various tissues, risks associated with genomic integration, and issues with tolerability. Founded in 2017, the company aims to expand the applications of gene therapy beyond traditional gene replacement, targeting a wide range of conditions including genetic disorders, ophthalmology, oncology, and metabolic diseases. By doing so, Ring Therapeutics seeks to provide healthcare solutions that allow for a broader array of therapeutic modalities and improved patient outcomes.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing therapeutic products and therapies for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts aim to address disabling symptoms in conditions affecting the nervous system, including Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma seeks to improve the quality of life and patient outcomes for individuals suffering from these rare conditions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Senti Biosciences is a clinical-stage biotechnology company specializing in synthetic biology and gene circuit engineering for therapeutic purposes. The company employs its Gene Circuits platform to develop innovative therapies that enhance precision in targeting cancer cells while avoiding damage to healthy cells. Senti Bio's approach involves the use of allogeneic chimeric antigen receptor natural killer (CAR-NK) cells that incorporate its gene circuit technologies, aimed at addressing various oncology indications. Among its product candidates are SENTI-202, a Logic Gated off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells without harming healthy bone marrow, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences aims to provide effective treatment options for difficult-to-treat diseases through advanced cell and gene therapies.

LifeMine Therapeutics, Inc. is a biotechnology company focused on discovering new drug modalities from eukaryotic microbes, particularly fungi. Founded in 2016 and headquartered in Cambridge, Massachusetts, with an additional office in New York, the company employs an integrated platform that combines genomics, artificial intelligence, and synthetic biology. This innovative Avatar-Rx platform utilizes high-throughput microbiology, data science, machine learning, genome engineering, and automation to explore the fungal biosphere for novel genetically encoded molecules with specific biological functions. By integrating chemoinformatic-assisted drug optimization and advanced chemical synthesis with biotransformation, LifeMine Therapeutics aims to advance a pipeline of new medicines into development.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing gene therapies through its innovative Anellovector platform. This platform leverages the biology of the human commensal virome to create redosable and targetable DNA therapies that address several limitations of existing gene therapies, such as restricted access to various tissues, risks associated with genomic integration, and issues with tolerability. Founded in 2017, the company aims to expand the applications of gene therapy beyond traditional gene replacement, targeting a wide range of conditions including genetic disorders, ophthalmology, oncology, and metabolic diseases. By doing so, Ring Therapeutics seeks to provide healthcare solutions that allow for a broader array of therapeutic modalities and improved patient outcomes.

Omega Therapeutics is a development-stage biotechnology company based in Cambridge, Massachusetts, focused on developing genomic medicines aimed at curing diseases. The company utilizes its proprietary epigenomic programming platform to create a new class of programmable epigenetic medicines, referred to as Omega Epigenomic Controllers. These controllers are designed to selectively modulate genomic activity, enabling precise tuning of the human genome to treat various medical conditions. Founded in 2016 and rebranded from VL42, Inc. in 2017, Omega Therapeutics is dedicated to transforming human medicine by harnessing the innate potential of the genome for therapeutic benefits.

LEXEO Therapeutics is a biotechnology company that specializes in the development of gene therapies, particularly those utilizing adeno-associated virus (AAV) technology. The company’s pipeline includes both rare and non-rare monogenic diseases, with a strong emphasis on hereditary and acquired conditions that present significant unmet medical needs. LEXEO's lead programs and preclinical candidates are primarily developed in collaboration with Weill Cornell Medicine’s Department of Genetic Medicine. The company aims to advance its clinical programs toward commercialization while maintaining an ongoing research partnership with Weill Cornell to enhance its preclinical efforts. Through its innovative approach, LEXEO Therapeutics seeks to transform the treatment landscape for diseases that impose a substantial burden on patients and society.

Ikena Oncology is a biotechnology company based in Boston, Massachusetts, focused on developing innovative therapies for cancer treatment. The company specializes in biomarker-driven and precision therapies that target specific mechanisms involved in cancer growth, spread, and resistance. Its product pipeline includes several candidates such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-930, an oral small-molecule inhibitor of the TEAD transcription factor in the Hippo signaling pathway. Additionally, Ikena is exploring a kynurenine-degrading enzyme with IK-412 and other discovery-stage programs targeting various oncogenic pathways. Established in 2016, Ikena Oncology aims to address unmet medical needs through a combination of chemistry, translational science, and patient-centered drug development.

Yumanity Therapeutics is transforming drug discovery for neurodegenerative diseases caused by protein misfolding. Founded in December 2014 by award-winning protein folding expert, Susan Lindquist, and renowned biotech industry leader, Tony Coles, Yumanity is working to identify and develop new, disease-modifying therapies that address several illnesses with critical unmet medical needs. The initial focus of the company is neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). The company’s proprietary platforms have already identified one potential new target for treating Parkinson’s disease, and Yumanity is actively advancing its new chemical lead series for this condition, as well as identifying additional compounds for Alzheimer’s disease and ALS.

Locanabio, Inc. is a biotechnology company based in San Diego, California, founded in 2016. It specializes in developing RNA-targeted gene therapies aimed at treating a range of underserved diseases, particularly in the areas of neuromuscular, neurodegenerative, and retinal conditions. The company's innovative platform focuses on modifying disease-causing RNA, allowing for the identification and correction of specific RNA sequences associated with rare genetic disorders. This approach distinguishes Locanabio's therapies from traditional DNA-targeted methods, offering a unique solution for patients with severe and challenging health issues. The company, which rebranded from Locana, Inc. in July 2020, is dedicated to advancing therapeutic candidates that address significant medical needs.

Noema Pharma AG is a biotechnology company based in Basel, Switzerland, founded in 2019. The company focuses on developing therapeutic products and therapies for orphan neurological disorders characterized by imbalanced neuronal networks. Its research and development efforts aim to address disabling symptoms in conditions affecting the nervous system, including Tuberous Sclerosis Complex, Trigeminal Neuralgia, and Tourette syndrome. By creating novel therapies, Noema Pharma seeks to improve the quality of life and patient outcomes for individuals suffering from these rare conditions.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Triplet Therapeutics, Inc. is focused on developing innovative therapies for repeat expansion disorders, which include conditions such as Huntington’s disease, myotonic dystrophy, and spinocerebellar ataxias. Founded in 2018 and based in Cambridge, Massachusetts, the company aims to address these disorders at their source by utilizing a unique approach that involves targeting the DNA Damage Response pathway through a single oligonucleotide. This biotherapeutic technology is designed to provide a comprehensive treatment solution for multiple repeat expansion disorders, enabling healthcare professionals to better manage these complex conditions.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

Foghorn Therapeutics is developing therapies based on a system that directs which genes our cells express, and when, where, and in what order.By manipulating this system with our unique Gene Traffic ControlTM platform, Foghorn will change how genes turn “on” and “off.” Unlike approaches that edit genes, this novel way of thinking will alter what our DNA has in store for us—and rewrite destiny for millions of people living with disease.With Gene Traffic ControlTM Foghorn is pioneering a new, major class of drug targets to develop unprecedented therapies for cancer and other serious diseases. We have already validated multiple targets, are in the process of developing drug candidates in multiple types of cancer and are beginning to explore other diseases.

KSQ Therapeutics, based in Cambridge, Massachusetts, specializes in precision functional genomics to discover innovative drug therapies. Founded in 2015, the company employs its proprietary CRISPRomics™ drug discovery engine to gain insights into the role of human genes across various diseases. This comprehensive understanding of disease biology allows KSQ to identify and validate high-confidence, patient-specific drug targets, enhancing the drug development process by focusing on candidates with the greatest therapeutic potential. The company has initiated several preclinical discovery programs aimed at developing novel medications that could significantly improve patient outcomes.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Moderna, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in developing therapeutics and vaccines using messenger RNA technology. Founded in 2010, the company focuses on various therapeutic areas, including infectious diseases, oncology, rare genetic disorders, and cardiovascular diseases. As of September 2024, Moderna has 40 mRNA development candidates in clinical development, demonstrating its commitment to advancing medical science. The company's mRNA platform has gained significant recognition, particularly with the authorization of its COVID-19 vaccine in December 2020. Moderna has established strategic collaborations with notable organizations such as AstraZeneca, Merck & Co., and the Bill & Melinda Gates Foundation. Additionally, it collaborates with Lonza Ltd. and Catalent for the manufacturing of its COVID-19 vaccine. Through its innovative approach, Moderna aims to provide effective solutions for a range of health challenges.

PrimeraDx, Inc. offers multiplexed infectious disease assays. It provides Scalable Target Amplification Routine technology, which is a method for simultaneous quantitative measurement of multiple target nucleic acids. The company also offers ViraQuant, a multiplexed assay providing quantitative and simultaneous measurement of viruses; and FungiQuant Yeast and FungiQuant Mold, which are fungal panels for mycology testing. Its products are used in miRNA, DNA methylation, quantitative genotyping, gene expression, and viral load measurement applications. PrimeraDx, Inc. was founded in 2004 and is based in Mansfield, Massachusetts.

PrimeraDx, Inc. offers multiplexed infectious disease assays. It provides Scalable Target Amplification Routine technology, which is a method for simultaneous quantitative measurement of multiple target nucleic acids. The company also offers ViraQuant, a multiplexed assay providing quantitative and simultaneous measurement of viruses; and FungiQuant Yeast and FungiQuant Mold, which are fungal panels for mycology testing. Its products are used in miRNA, DNA methylation, quantitative genotyping, gene expression, and viral load measurement applications. PrimeraDx, Inc. was founded in 2004 and is based in Mansfield, Massachusetts.

Standard BioTools, previously known as Fluidigm, is driven by a bold vision – unleashing tools to accelerate breakthroughs in human health. Standard BioTools has an established portfolio of essential, standardized next-generation technologies that help biomedical researchers develop medicines faster and better. As a leading solutions provider, the Company provides reliable and repeatable insights in health and disease using its proprietary mass cytometry and microfluidics technologies that help transform scientific discoveries into better patient outcomes. Standard BioTools works with leading academic, government, pharmaceutical, biotechnology, plant and animal research, and clinical laboratories worldwide, focusing on the most pressing needs in translational and clinical research, including oncology, immunology, and immunotherapy.